DAYBREAK: A Study of Setmelanotide in Patients With Specific Gene Variants in the MC4R Pathway
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| ClinicalTrials.gov Identifier: NCT04963231 |
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Recruitment Status :
Recruiting
First Posted : July 15, 2021
Last Update Posted : November 30, 2022
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Sponsor:
Rhythm Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Rhythm Pharmaceuticals, Inc.
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Brief Summary:
A 2-stage (open-label run-in, followed by randomized, double-blind, placebo-controlled withdrawal) trial of setmelanotide in patients with obesity and specific gene defects variants in the melanocortin-4 receptor (MC4R) pathway.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Genetic Obesity | Drug: Setmelanotide Drug: Placebo | Phase 2 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 150 participants |
| Allocation: | Randomized |
| Intervention Model: | Sequential Assignment |
| Masking: | Triple (Participant, Care Provider, Investigator) |
| Masking Description: | Stage 1 of the trial is open-label. Patients who are eligible to enter Stage 2 of the trial will be randomized in a blinded manner at the Stage 2 Entry Visit in a 2:1 ratio to receive either setmelanotide (2) or placebo (1). |
| Primary Purpose: | Treatment |
| Official Title: | A Phase 2, Two-Stage (Open-Label Run-in Followed by Randomized Withdrawal), Double-Blind, Placebo-Controlled Study of Setmelanotide in Patients With Specific Gene Variants in the Melanocortin-4 Receptor Pathway |
| Actual Study Start Date : | November 30, 2021 |
| Estimated Primary Completion Date : | October 2024 |
| Estimated Study Completion Date : | October 2024 |
Resource links provided by the National Library of Medicine
MedlinePlus related topics:
Genes and Gene Therapy
Drug Information available for:
Setmelanotide
| Arm | Intervention/treatment |
|---|---|
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Experimental: Setmelanotide
Patients with specific gene variants in the MC4R pathway on setmelanotide
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Drug: Setmelanotide
Subcutaneous injection |
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Placebo Comparator: Placebo
Patients with specific gene variants in the MC4R pathway on placebo
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Drug: Placebo
Subcutaneous injection |
Primary Outcome Measures :
- The proportion of patients by genotype who achieve MC4R pathway who achieve a ≥5% reduction in BMI [ Time Frame: Baseline to End of Stage 1 (16 weeks) ]To evaluate the proportion of patients with obesity and genetic variants in the MC4R pathway who achieve a clinically meaningful reduction in body weight in response to setmelanotide
Secondary Outcome Measures :
- Mean change and percent change in BMI in all patients and patients ≥18 years old, per gene [ Time Frame: Baseline to End of Stage 1 (16 weeks) ]To evaluate change in weight parameters in response to setmelanotide in patients with genetic variants in a specific gene in the MC4R pathway at the end of open-label treatment
- Mean change and percent change in body weight in patients ≥18 years old, per gene [ Time Frame: Baseline to End of Stage 1 (16 weeks) ]To evaluate change in weight parameters in response to setmelanotide in patients with genetic variants in a specific gene in the MC4R pathway at the end of open-label treatment
- Mean change in BMI Z-score in patients <18 years old, per gene [ Time Frame: Baseline to End of Stage 1 (16 weeks) ]To evaluate change in weight parameters in patients less than 18 years of age in response to setmelanotide in patients with genetic variants in a specific gene in the MC4R pathway at the end of open-label treatment
- Mean percent change in the weekly average of the daily maximal hunger score in patients ≥12 years old, per gene [ Time Frame: Baseline to End of Stage 1 (16 weeks) ]To evaluate change in hunger in response to setmelanotide in patients ≥12 years old with genetic variants in a specific gene in the MC4R pathway at the end of open-label treatment
- The proportion of patients ≥12 years old, per gene, who achieve a ≥2 point reduction in the weekly average of the daily maximal hunger score [ Time Frame: Baseline to End of Stage 1 (16 weeks) ]The proportion of patients ≥12 years old, per gene, who achieve a ≥2 point reduction (improvement) in the weekly average of the daily maximal hunger score
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| Ages Eligible for Study: | 6 Years to 65 Years (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Patients must have a pre-identified genetic variant in an established MC4R pathway gene that contributes to obesity
- Age 6 to 65 years, inclusive
- Obesity, defined as BMI ≥40 kg/m2 for patients≥18 years of age or BMI ≥97th percentile for age and gender for patients 6 up to 17 years of age
- Study participant and/or parent or guardian is able to understand all study procedures and provide consent/assent
- Use of highly effective contraception
- Symptoms or behaviors of hyperphagia
Exclusion Criteria:
- Patients with the following genetic variants: biallelic Bardet-Biedl Syndrome (BBS); biallelic Alström Syndrome 1 (ALMS1); homozygous, heterozygous, or compound heterozygous variants in MC4R, POMC, PCSK1, LEPR, nuclear receptor coactivator 1 (NCOA1; steroid receptor coactivator-1 [SRC1]) or SRC homology 2 B adapter protein 1 (SH2B1) genes as well as 16p11.2 chromosomal deletions that include the SH2B1 gene
- Recent intensive diet and/or exercise regimen with or without the use of weight loss agents including herbal medications that has resulted in weight loss >2% within previous 3 months
- Bariatric surgery within the previous 6 months.
- Documented diagnosis of schizophrenia, bipolar disorder, personality disorder, major depressive disorder, or other psychiatric disorder(s)
- Any suicidal ideation of type 4 or 5 on the Columbia-Suicide Severity Rating Scale (C-SSRS) or Patient Health Questionnaire 9 (PHQ 9) score of ≥15 during Screening, any suicide attempt in patient's lifetime years, or any suicidal behavior in the last month.
- Current, clinically significant pulmonary, cardiac, or oncologic disease considered severe enough to interfere with the study
- Has significant features of (or meets the diagnostic criteria for) a genetic syndrome that is associated with obesity
- HbA1C >10.0% at Screening
- History of significant liver disease
- Glomerular filtration rate (GFR) <30 mL/min at Screening
- History or close family history of melanoma or patient history of oculocutaneous albinism
- Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions
- Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing
- Patients previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide
- Significant hypersensitivity to any excipient in the study drug
- Females who are breastfeeding or nursing
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04963231
Contacts
| Contact: Rhythm Clinical Trials | (857) 264-4280 | clinicaltrials@rhythmtx.com | |
| Contact: Physician Inquiry: Clinical Trials | (857) 264-4280 | clinicaltrials@rhythmtx.com |
Locations
Show 32 study locations
Sponsors and Collaborators
Rhythm Pharmaceuticals, Inc.
Investigators
| Study Chair: | David Meeker, MD | Rhythm Pharmaceuticals, Inc. |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Rhythm Pharmaceuticals, Inc. |
| ClinicalTrials.gov Identifier: | NCT04963231 |
| Other Study ID Numbers: |
RM-493-034 |
| First Posted: | July 15, 2021 Key Record Dates |
| Last Update Posted: | November 30, 2022 |
| Last Verified: | November 2022 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
| Product Manufactured in and Exported from the U.S.: | Yes |
Keywords provided by Rhythm Pharmaceuticals, Inc.:
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Melanocortin-4 Receptor Genetic Obesity Leptin |
Additional relevant MeSH terms:
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Obesity Overweight Overnutrition Nutrition Disorders Body Weight |